Cutaneous LupusPhase 1/2NCT03288324
Open-label Study of Tofacitinib for Moderate to Severe Skin Involvement in Young Adults With Lupus
A Phase 1/2 study testing Tofacitinib in people with Cutaneous Lupus and Systemic Lupus Erythematosus, run by Children's Hospital Medical Center, Cincinnati at 2 sites in United States.
Trial summary
This 76-week, 3-part Phase 1b/2 study is intended to evaluate the pharmacological properties (pharmacokinetics and pharmacodynamics), safety, tolerability and preliminary effectiveness of TOFA administrated to young adults (18-45 years) with moderately to severely active SLE-CL. Read more about this study
- Who is it for?
- Ages 18 to 45 years
- Conditions the study is for
- Cutaneous Lupus, Systemic Lupus Erythematosus
- What is being tested?
- Tofacitinib
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- 2 sitesUnited States
- Study dates
- 23 Aug 2017to 1 Dec 2023
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 45 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Male or female > 18 years of age and < 45 years of age and > 40 kg body weight.
- Fulfilled at least 4 out of the 11 Classification Criteria for SLE by the time of screening.
- Willing to give written informed consent, must fully understand the requirements of the trial, and must be willing to comply with all trial visits and assessments.
- CLASI activity score of 8 or higher at screening and baseline despite standard of care therapy.
- Stable dose of prednisone of ≤ 20 mg/day within 2 weeks of enrollment.
- Female subjects of childbearing potential must use a highly effective method of contraception to prevent pregnancy (abstinence is considered highly effective) and must agree to continue to practice adequate contraception for the duration of their participation in the trial and for 28 days after their last dose of TOFA.
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- Female subjects of childbearing potential must have a negative serum pregnancy test at screening and a negative urine pregnancy test at Trial Day 1 before dosing.
- For subjects receiving leflunomide treatment, total daily dose does not exceed 20 mg.
- A negative QuantiFERON-TB Gold In-Tube test performed within the 3 months prior to screening, or within the screening period prior to baseline. A negative PPD test can be substituted for the QuantiFERON-TB.
- Subjects either have protective varicella titers or evidence of having been vaccinated against varicella.
Who cannot join
- Mild SLE-CL defined as a CLASI activity score of 7 or lower at screening and baseline.
- Increase in CS dosing within 2 weeks prior to Trial Day 1, or expected to require an increase in CS dosing during the first 4 weeks of the study.
- Use of i.v. corticosteroids within 4 weeks prior to Trial Day 1.
- Increase in dosing of methotrexate, leflunomide, within 4 weeks before Trial Day 1 or expected to require an increase during the first 8-weeks of the study.
- Increase in dosing of hydroxychloroquine, or chloroquine within 4 weeks before Trial Day 1 or expected to require an increase during the first 8-weeks of the study.
- Rituximab within 1 year of Trial Day 1.
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- Increase in dosing of any medication or herbal treatment considered to have immunosuppressive properties with 4 weeks before Trial Day 1.
- Prior treatment with or known intolerability of TOFA.
- Use of cyclophosphamide (i.v. or oral), cyclosporine, or tacrolimus within 12 weeks prior to Trial Day 1.
- Treatment with other investigational agents within the last 6 months or 5 half-lives, or as per washout requirement from the previous protocol, whichever is longer.
- Estimated glomerular filtration rate less than or equal to 60 mL/min /1.73 m2.
- Known positive Human Immunodeficiency Virus (HIV), Hepatitis C Virus (HCV), or Hepatitis B surface antigen (HBsAg) serology.
- Any condition, including findings in the laboratory tests, medical history, or other screening assessments, that, in the opinion of the Investigator, constitutes an inappropriate risk or a contraindication for participation in the trial or that could interfere with the trial's objectives, conduct, or evaluation.
- Active central nervous system SLE deemed to be severe or progressive and/or associated with significant cognitive impairment leading to inability to provide informed consent and/or comply with the protocol.
- Significant renal disease due to a reason(s) other than Lupus Nephritis (e.g. diabetes mellitus, renovascular disease, or antiphospholipid syndrome).
- Severely active Lupus Nephritis defined as a renal BILAG A score.
- History of dialysis within 3 months prior to Trial Day 1 or expected to need during the trial.
- History of or planned renal or other organ transplantation.
- Known active clinically significant viral, bacterial or fungal infection, or any major episode of infection requiring hospitalization or treatment with parenteral anti-infectives within 8 weeks of screening, or completion of oral anti-infectives within 2 weeks of Trial Day 1.
- Breastfeeding or currently pregnant.
- Legal incapacity or limited legal capacity to provide informed consent or assent.
- Blood dyscrasias, including:
- Hgb <10 g/dL or Hct <33%.
- WBC <3.0 x 109/L.
- Neutrophil count <1.2 x 109/L.
- Platelet count <100 x 109/L.
- Lymphocyte count of <0.5 x 109/L.
- AST or ALT > 1.5 times the upper limit of normal or any other clinically significant laboratory abnormality.
- History of any other rheumatic autoimmune disease.
- Infections:
- Latent or active TB or any history of previous TB.
- Chronic infections.
- Any infection requiring hospitalization, parenteral antimicrobial therapy or judged to be opportunistic by the investigator within the 6 months prior to the first dose of study drug.
- Any treated infections within 2 weeks.
- History of recurrent (more than one episode) herpes zoster or disseminated (a single episode) herpes zoster or disseminated (a single episode) herpes simplex.
- History or current symptoms suggestive of any lymphoproliferative disorder, including Cytomegaly Virus (CMV) or Epstein Barr Virus (EBV) related lymphoproliferative disorder, history of lymphoma, leukemia, or signs and symptoms suggestive of current lymphatic disease.
- Subjects taking potent and moderate cytochrome P450 3A4 (CYP3A4) inhibitors (see Appendix 2).
- Subjects taking potent and moderate CYP3A4 inducers (see Appendix 2).
- Subjects who have been vaccinated with live or attenuated vaccines within the 6 weeks prior to the first dose of study medication. All subjects should be up-to-date with respect to standard of care vaccinations (as defined by their country health ministry) as permitted by past immunosuppressive therapy for SLE.
- Subjects with a malignancy or with a history of malignancy with the exception of adequately treated or excised non-metastatic basal cell or squamous cell cancer of the skin or cervical carcinoma in situ.
- Subjects with a history or current diagnosis of diverticulitis.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Cincinnati Childrens Hospital Medical Center
Cincinnati, Ohio, 45229
MetroHealth Medical Center
Cleveland, Ohio, 44109
About this study
Official title: A 3-part Open-label Study Assessing Safety, Tolerability, Pharmacokinetic and -Dynamic Profiles, and Efficacy of Tofacitinib in Young Adults From Age 18 to 45 With Moderate to Severe Skin Involvement Due to Lupus
This 76-week, 3-part Phase 1b/2 study is intended to evaluate the pharmacological properties (pharmacokinetics and pharmacodynamics), safety, tolerability and preliminary effectiveness of TOFA administrated to young adults (18-45 years) with moderately to severely active SLE-CL. Subjects will be studied at the Cincinnati Children's Hospital Medical Center (CCHMC) and in Cleveland at MetroHealth Medical Center.
Read the detailed description
Cohort 1 (n=10, weight > 40kg and age > 18 years and ≤ 45 years ) will undergo intense PK-sampling to determine exposures following TOFA dosed at 5 mg BID. TOFA dose escalation will not be considered for inadequate response of SLE-CL.
Cohort 2 (n=10, weight > 40kg and age > 18 years and ≤ 45 years) will be treated with the same dose as Cohort 1. No PK sampling will occur for Cohort 2. Enrollment of Cohort 2 will only start once Cohort 1 has completed 8 weeks of TOFA and results of PK analyses from Cohort 1 are available.
- Part A (up to week 8) requires stable background medications;
- Part B (up to week 24) allows for tapering of corticosteroids (CS) in the setting of significant clinical improvement of SLE-CL as defined by a decrease in Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI) activity score by >50% from baseline , and
- Part C (until week 76) permits tapering of other background medications in subjects with clinical remission of SLE-CL (CLASI activity score=0). TOFA dosing is kept stable during Part C.
- Conditions
- Cutaneous Lupus, Systemic Lupus Erythematosus
- Being tested
- Tofacitinib
- Medical subject headings
- Lupus Erythematosus, Systemic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TofacitinibDrug
Also called Xeljanz
Tofacitinib 5 mg twice daily
| Study group | Role | What they receive |
|---|---|---|
| Tofacitinib Arm | Experimental | open-label study Receives: Drug: Tofacitinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Oral Clearance (CL/F) (Cohort 1 Only)
Apparent total clearance of the drug from plasma after oral administration
Measured: Day 5
Secondary outcomes (7)
Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI) Activity Score
Proportion of subjects who achieve a skin response per the validated CLASI
The Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI) activity score consists of two scores. The first summarizes the activity of the disease while the second is a measure of the damage done by the disease. The Activity Score range is 0-70 with the maximum score (70) indicating the worst outcome. The Damage Score range is 0-80 with the maximum score (80) indicating the worst outcome.
Measured: weeks 4, 8 and 24 compared to baseline.
AUCt (Cohort 1 Only)
Area under the plasma concentration-time curve linear scale Median Concentration (ng/mL) per nominal time 0-8 hours.
Measured: Day 5
Cmax (Cohort 1 Only)
Maximum (or peak) plasma concentration of Tofacitinib
Measured: Day 5
Tmax (Cohort 1 Only)
Time to reach maximum (peak) plasma concentration following administration of Tofacitinib
Measured: Day 5
Vz/F (Cohort 1 Only)
Apparent volume of distribution during terminal phase after non-intravenous administration
Measured: Day 5
Half-life of Tofacitinib (Cohort 1 Only)
half-life of Tofacitinib
Measured: Day 5
Safety of Tofacitinib: Total Number of Adverse Events is Reported (Cohorts 1 and 2)
Rate and severity of adverse events and lab abnormalities experienced by participants in both cohorts of the research study.
Measured: 76 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Sep 2017
- Study start23 Aug 2017
- Primary completion1 Nov 2022
- Study completion1 Dec 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Cutaneous LupusStudies of the same condition, open and under way.
- More trials run by Children's Hospital Medical Center, CincinnatiThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Sponsor type
- University, hospital or other
- Collaborators
- Pfizer
- Responsible party
- Sponsor
- Investigators
- Hermine Brunner, MD, Principal investigator, Cincinnati Childrens Hospital Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03288324
- Sponsor's protocol ID
- WI211648
- Status last verified
- Feb 2026
- First submitted
- 29 Aug 2017
- Last updated
- 3 Mar 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A 3-part Open-label Study Assessing Safety, Tolerability, Pharmacokinetic and -Dynamic Profiles, and Efficacy of Tofacitinib in Young Adults From Age 18 to 45 With Moderate to Severe Skin Involvement Due to Lupus
- ClinicalTrials.gov ID
- NCT03288324
- Acronym / sponsor ID
- WI211648
- Conditions
- Cutaneous Lupus, Systemic Lupus Erythematosus
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 13 participants (actual)
- Lead sponsor
- Children's Hospital Medical Center, CincinnatiUniversity, hospital or other
- Collaborators
- Pfizer
- Study start
- 23 Aug 2017 (actual)
- Primary completion
- 1 Nov 2022 (actual)
- Study completion
- 1 Dec 2023 (actual)
- First posted
- 20 Sep 2017 (actual)
- Results
- Posted 30 Jul 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This 76-week, 3-part Phase 1b/2 study is intended to evaluate the pharmacological properties (pharmacokinetics and pharmacodynamics), safety, tolerability and preliminary effectiveness of TOFA administrated to young adults (18-45 years) with moderately to severely active SLE-CL. Subjects will be studied at the Cincinnati Children's Hospital Medical Center (CCHMC) and in Cleveland at MetroHealth Medical Center.
Detailed description
Cohort 1 (n=10, weight > 40kg and age > 18 years and ≤ 45 years ) will undergo intense PK-sampling to determine exposures following TOFA dosed at 5 mg BID. TOFA dose escalation will not be considered for inadequate response of SLE-CL.
Cohort 2 (n=10, weight > 40kg and age > 18 years and ≤ 45 years) will be treated with the same dose as Cohort 1. No PK sampling will occur for Cohort 2. Enrollment of Cohort 2 will only start once Cohort 1 has completed 8 weeks of TOFA and results of PK analyses from Cohort 1 are available.
- Part A (up to week 8) requires stable background medications;
- Part B (up to week 24) allows for tapering of corticosteroids (CS) in the setting of significant clinical improvement of SLE-CL as defined by a decrease in Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI) activity score by >50% from baseline , and
- Part C (until week 76) permits tapering of other background medications in subjects with clinical remission of SLE-CL (CLASI activity score=0). TOFA dosing is kept stable during Part C.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Tofacitinib Arm | Experimental | Drug: Tofacitinibopen-label study |
Who may qualify
- Ages
- 18 to 45 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (10 listed)
- Male or female > 18 years of age and < 45 years of age and > 40 kg body weight.
- Fulfilled at least 4 out of the 11 Classification Criteria for SLE by the time of screening.
- Willing to give written informed consent, must fully understand the requirements of the trial, and must be willing to comply with all trial visits and assessments.
- CLASI activity score of 8 or higher at screening and baseline despite standard of care therapy.
- Stable dose of prednisone of ≤ 20 mg/day within 2 weeks of enrollment.
Key exclusion criteria (41 listed)
- Mild SLE-CL defined as a CLASI activity score of 7 or lower at screening and baseline.
- Increase in CS dosing within 2 weeks prior to Trial Day 1, or expected to require an increase in CS dosing during the first 4 weeks of the study.
- Use of i.v. corticosteroids within 4 weeks prior to Trial Day 1.
- Increase in dosing of methotrexate, leflunomide, within 4 weeks before Trial Day 1 or expected to require an increase during the first 8-weeks of the study.
- Increase in dosing of hydroxychloroquine, or chloroquine within 4 weeks before Trial Day 1 or expected to require an increase during the first 8-weeks of the study.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TofacitinibAlso: XeljanzTofacitinib 5 mg twice daily | Drug | Tofacitinib Arm |
Primary outcome
Oral Clearance (CL/F) (Cohort 1 Only)
Time frame: Day 5
Apparent total clearance of the drug from plasma after oral administration
7 secondary outcomes
Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI) Activity Score
Time frame: weeks 4, 8 and 24 compared to baseline.
AUCt (Cohort 1 Only)
Time frame: Day 5
Cmax (Cohort 1 Only)
Time frame: Day 5
Tmax (Cohort 1 Only)
Time frame: Day 5
Vz/F (Cohort 1 Only)
Time frame: Day 5
Half-life of Tofacitinib (Cohort 1 Only)
Time frame: Day 5
Safety of Tofacitinib: Total Number of Adverse Events is Reported (Cohorts 1 and 2)
Time frame: 76 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Mar 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Hermine Brunner, MD principal investigator
Cincinnati Childrens Hospital Medical Center
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Tofacitinib in people with Cutaneous Lupus and Systemic Lupus Erythematosus, run by Children's Hospital Medical Center, Cincinnati at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 41 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 3 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 30 Jul 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Oral Clearance (CL/F) (Cohort 1 Only) (time frame: Day 5). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Mar 2026.